Talking Rheumatology Spotlight
Sep 1, 2026 · 58 min · 11 segments
Dr Lilase Hakoun, paediatric rheumatology trainee, talks with Dr Charalampia Papadopoulou about JDM. They explore together a rare but important condition. Approaches to diagnosis and evidence based…
Charalampia PapadopoulouGuest
Lilase HakounHost
When we talk about future therapies in JDM, what is genuinely exciting and what needs to be framed more cautiously?
So some things are exciting because they are close enough to clinic to change practice soon.
And then there are other things that they are exciting because they expand the horizon.
But we need to understand that they remain experimental and they should be presented that way.
We are doing a smarter use of the MSA and the biomarkers, and we are having stronger evidence about JAK inhibitors.
And then in the more frontier category, we have the CAR T therapies, which are clearly attracting an enormous interest.
There are now early reports and reviews suggesting that CAR-T approaches may induce profound remission in refractory juvenile autoimmune diseases, including severe JADM, by effectively resetting pathogenic B-cell-driven immunity.
There is also growing trial interest relevant to juvenile myositis, But having said that, at the same time, we have to be cautious.
The experience remains limited, and we still need to define the long-term safety, the costs, And some of the major questions is which patients should be selected, what is the toxicity, and where these therapies will fit in pediatric autoimmune diseases.
So CAR-T is a very exciting proof of concept for the most refractory end of the disease.
But definitely at the moment, at least, not something that is about to replace the current care.
So, Dr. Harris, if you wanted listeners to take away just a handful of messages from this episode, what would they be?
And then I would point three, you know, points that I would like them to take away.
You need to understand the phenotype before because that way you will understand the patient better.
Secondly, vasculopathy is central to the disease story, especially in the most severe phenotypes.
When we talk about future therapies in JDM, what is genuinely exciting and what needs to be framed more cautiously?
So some things are exciting because they are close enough to clinic to change practice soon.
And then there are other things that they are exciting because they expand the horizon.
But we need to understand that they remain experimental and they should be presented that way.
We are doing a smarter use of the MSA and the biomarkers, and we are having stronger evidence about JAK inhibitors.
And then in the more frontier category, we have the CAR T therapies, which are clearly attracting an enormous interest.
There are now early reports and reviews suggesting that CAR-T approaches may induce profound remission in refractory juvenile autoimmune diseases, including severe JADM, by effectively resetting pathogenic B-cell-driven immunity.
There is also growing trial interest relevant to juvenile myositis, But having said that, at the same time, we have to be cautious.
The experience remains limited, and we still need to define the long-term safety, the costs, And some of the major questions is which patients should be selected, what is the toxicity, and where these therapies will fit in pediatric autoimmune diseases.
So CAR-T is a very exciting proof of concept for the most refractory end of the disease.
But definitely at the moment, at least, not something that is about to replace the current care.
So, Dr. Harris, if you wanted listeners to take away just a handful of messages from this episode, what would they be?
And then I would point three, you know, points that I would like them to take away.
You need to understand the phenotype before because that way you will understand the patient better.
Secondly, vasculopathy is central to the disease story, especially in the most severe phenotypes.
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